IL282191A - שיטות ותכשירים לעריכת רנ" א - Google Patents

שיטות ותכשירים לעריכת רנ" א

Info

Publication number
IL282191A
IL282191A IL282191A IL28219121A IL282191A IL 282191 A IL282191 A IL 282191A IL 282191 A IL282191 A IL 282191A IL 28219121 A IL28219121 A IL 28219121A IL 282191 A IL282191 A IL 282191A
Authority
IL
Israel
Prior art keywords
compositions
methods
rnas
editing
editing rnas
Prior art date
Application number
IL282191A
Other languages
English (en)
Original Assignee
Univ Beijing
Edigene Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Beijing, Edigene Inc filed Critical Univ Beijing
Publication of IL282191A publication Critical patent/IL282191A/he

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    • CCHEMISTRY; METALLURGY
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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0636T lymphocytes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/34Allele or polymorphism specific uses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00Genetically modified cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/10Plasmid DNA
    • C12N2800/106Plasmid DNA for vertebrates
    • C12N2800/107Plasmid DNA for vertebrates for mammalian

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Virology (AREA)
  • Cardiology (AREA)
  • Hematology (AREA)
  • Neurosurgery (AREA)
  • Diabetes (AREA)
  • Hospice & Palliative Care (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Neurology (AREA)
  • Cell Biology (AREA)
IL282191A 2018-10-12 2021-04-08 שיטות ותכשירים לעריכת רנ" א IL282191A (he)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2018110105 2018-10-12
CN2019082713 2019-04-15
PCT/CN2019/110782 WO2020074001A1 (en) 2018-10-12 2019-10-12 Methods and Compositions for Editing RNAs

Publications (1)

Publication Number Publication Date
IL282191A true IL282191A (he) 2021-05-31

Family

ID=70163607

Family Applications (1)

Application Number Title Priority Date Filing Date
IL282191A IL282191A (he) 2018-10-12 2021-04-08 שיטות ותכשירים לעריכת רנ" א

Country Status (20)

Country Link
US (2) US20210355494A1 (he)
EP (2) EP3864152B1 (he)
JP (2) JP7252328B2 (he)
CN (4) CN116218836A (he)
AU (2) AU2019357450B2 (he)
BR (1) BR112021006844A8 (he)
CA (1) CA3115864A1 (he)
CL (1) CL2021000880A1 (he)
CO (1) CO2021005956A2 (he)
CR (1) CR20210243A (he)
DK (1) DK3864152T3 (he)
EC (1) ECSP21033637A (he)
ES (1) ES2962434T3 (he)
IL (1) IL282191A (he)
MX (1) MX2021004187A (he)
PE (1) PE20211402A1 (he)
PH (1) PH12021550805A1 (he)
SG (1) SG11202103666PA (he)
TW (1) TW202028466A (he)
WO (1) WO2020074001A1 (he)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11932856B2 (en) 2017-03-03 2024-03-19 The Regents Of The University Of California RNA targeting of mutations via suppressor tRNAs and deaminases
US20220307020A1 (en) 2019-04-15 2022-09-29 Edigene Inc. Methods and compositions for editing rnas
CR20220063A (es) 2019-07-12 2022-07-22 Univ Beijing Edición de ácido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actúa sobre ácido ribonucleico endógeno (adar) utilizando ácidos ribonucleicos (arn) modificados genéticamente
CN115038789A (zh) 2019-12-02 2022-09-09 塑造治疗公司 治疗性编辑
WO2021136404A1 (zh) 2019-12-30 2021-07-08 博雅辑因(北京)生物科技有限公司 一种治疗Usher综合征的方法和其组合物
JP2023509179A (ja) 2019-12-30 2023-03-07 北京▲輯▼因医▲療▼科技有限公司 Leaper技術に基づくmps ihの治療方法及び組成物
EP4137161A4 (en) * 2020-04-15 2023-10-11 EdiGene Therapeutics (Beijing) Inc. METHOD AND MEDICATION FOR THE TREATMENT OF HURLER SYNDROME
AU2021279027A1 (en) * 2020-05-28 2022-12-08 Korro Bio, Inc. Methods and compositions for the ADAR-mediated editing of SERPINA1
CN113897359A (zh) * 2020-07-06 2022-01-07 博雅辑因(北京)生物科技有限公司 一种改善的rna编辑方法
EP4177345A1 (en) 2020-07-06 2023-05-10 EdiGene Therapeutics (Beijing) Inc. Improved rna editing method
IL278401A (he) 2020-10-29 2022-05-01 Yeda Res & Dev פולינוקלאוטידים לעריכה של רנ" א ושיטה לשימוש בהם
EP4240338A1 (en) * 2021-01-04 2023-09-13 The Regents of The University of California <smallcaps/>? ? ?in vivo? ? ? ? ?programmable rna editingvia recruitment of endogenous adars
CN117015605A (zh) * 2021-01-12 2023-11-07 北京大学 使用工程化rna通过利用内源性adar进行靶向rna编辑
US20230123513A1 (en) * 2021-06-15 2023-04-20 Massachusetts Institute Of Technology Deaminase-based rna sensors
WO2023278436A1 (en) * 2021-06-29 2023-01-05 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
WO2023020574A1 (en) * 2021-08-18 2023-02-23 Peking University Engineered adar-recruiting rnas and methods of use thereof
WO2023060186A1 (en) 2021-10-07 2023-04-13 Hepatx Corporation Methods of tracking donor cells in a recipient
CA3236182A1 (en) * 2021-10-29 2023-05-04 Z. Josh Huang Compositions and systems for rna-programable cell editing and methods of making and using same
WO2023143539A1 (en) * 2022-01-28 2023-08-03 Edigene Therapeutics (Beijing) Inc. Engineered adar-recruiting rnas and methods of use thereof
WO2023185231A1 (en) * 2022-04-02 2023-10-05 Edigene Therapeutics (Beijing) Inc. Engineered adar-recruiting rnas and methods of use for usher syndrome
WO2024086650A1 (en) * 2022-10-18 2024-04-25 Locanabio, Inc. Compositions and methods comprising programmable snrnas for rna editing

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016094845A2 (en) 2014-12-12 2016-06-16 Woolf Tod M Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
WO2016097212A1 (en) * 2014-12-17 2016-06-23 Proqr Therapeutics Ii B.V. Targeted rna editing
JP6624743B2 (ja) 2015-07-14 2019-12-25 学校法人福岡大学 部位特異的rna変異導入方法およびそれに使用する標的編集ガイドrnaならびに標的rna−標的編集ガイドrna複合体
DE102015012522B3 (de) * 2015-09-26 2016-06-02 Eberhard Karls Universität Tübingen Verfahren und Substanzen zur gerichteten RNA-Editierung
WO2017220751A1 (en) * 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
PL3507366T3 (pl) * 2016-09-01 2021-05-04 Proqr Therapeutics Ii B.V. Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rna
WO2018134301A1 (en) 2017-01-19 2018-07-26 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in rna editing
US11932856B2 (en) 2017-03-03 2024-03-19 The Regents Of The University Of California RNA targeting of mutations via suppressor tRNAs and deaminases
US11739308B2 (en) 2017-03-15 2023-08-29 The Broad Institute, Inc. Cas13b orthologues CRISPR enzymes and systems

Also Published As

Publication number Publication date
AU2023202184A1 (en) 2023-05-04
CN115651927B (zh) 2023-06-16
CL2021000880A1 (es) 2021-12-10
JP2023078373A (ja) 2023-06-06
EP4306116A2 (en) 2024-01-17
US20210355494A1 (en) 2021-11-18
WO2020074001A1 (en) 2020-04-16
CA3115864A1 (en) 2020-04-16
CN113631708B (zh) 2022-05-24
AU2019357450B2 (en) 2023-01-12
JP2021534810A (ja) 2021-12-16
CO2021005956A2 (es) 2021-07-30
DK3864152T3 (da) 2023-10-09
BR112021006844A8 (pt) 2023-03-21
EP3864152B1 (en) 2023-08-16
BR112021006844A2 (pt) 2021-07-20
PH12021550805A1 (en) 2021-10-04
EP3864152A4 (en) 2021-10-13
PE20211402A1 (es) 2021-08-02
MX2021004187A (es) 2021-09-08
US20210310026A1 (en) 2021-10-07
SG11202103666PA (en) 2021-05-28
CN113631708A (zh) 2021-11-09
KR20210076082A (ko) 2021-06-23
CN116218836A (zh) 2023-06-06
ES2962434T3 (es) 2024-03-19
CN115651927A (zh) 2023-01-31
CR20210243A (es) 2021-10-07
EP3864152A1 (en) 2021-08-18
AU2019357450A1 (en) 2021-06-03
CN116042611A (zh) 2023-05-02
EP4306116A3 (en) 2024-04-17
TW202028466A (zh) 2020-08-01
JP7252328B2 (ja) 2023-04-04
ECSP21033637A (es) 2021-08-31

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